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Journal Articles

Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials

Blood (2022) 140 (Supplement 1): 5355–5357.

DOI: https://doi.org/10.1182/blood-2022-162400

Published: 2022

Journal Articles

Return Visit Rates after an Emergency Department Treat-and-Release Visit for Children with Sickle Cell Pain Episodes

Blood (2022) 140 (Supplement 1): 5418–5419.

DOI: https://doi.org/10.1182/blood-2022-166132

Published: 2022

Journal Articles

Lovo-cel (bb1111) Gene Therapy for Sickle Cell Disease: Updated Clinical Results and Investigations into Two Cases of Anemia from Group C of the Phase 1/2 HGB-206 Study

Mark C. Walters, Alexis A. Thompson, Janet L. Kwiatkowski, Suhag Parikh, Markus Y. Mapara, Stacey Rifkin-Zenenberg, Banu Aygun, Kimberly A. Kasow, Alex Miller, Lixin Zhang, Anjulika Chawla, Elizabeth R. Macari, Francis J. Pierciey, Jr., John F. Tisdale, Julie Kanter

Blood (2022) 140 (Supplement 1): 26–28.

DOI: https://doi.org/10.1182/blood-2022-162288

Published: 2022

Journal Articles

Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia

Franco Locatelli, Mark C. Walters, Janet L. Kwiatkowski, Marina Cavazzana, Suradej Hongeng, John B. Porter, Adrian J. Thrasher, Andreas E. Kulozik, Isabelle Thuret, John E.J. Rasko, Evangelia Yannaki, Martin G. Sauer, Shamshad Ali, Himal Thakar, Katiana Gruppioni, Alexis A. Thompson

Blood (2022) 140 (Supplement 1): 8200–8202.

DOI: https://doi.org/10.1182/blood-2022-163178

Published: 2022

Journal Articles

Optimizing Shared Decision Making about Hydroxyurea in Young Children with Sickle Cell Anemia

Blood (2022) 140 (Supplement 1): 10857–10859.

DOI: https://doi.org/10.1182/blood-2022-167914

Published: 2022

Journal Articles

Relationship of Hydroxyurea Adherence to Depression and Fatigue Among Children and Adolescents with Sickle Cell Disease: A Longitudinal Cohort Study

Sherif M. Badawy, Kathryn King, Olivia E. Atherton, Daniel Mroczek, Alexis A. Thompson, David Cella, Tonya M. Palermo, Jane L. Holl, Robert I. Liem

Blood (2021) 138 (Supplement 1): 3104.

DOI: https://doi.org/10.1182/blood-2021-149902

Published: 2021

Journal Articles

Improvement in Health-Related Quality of Life Following Treatment with Betibeglogene Autotemcel in Patients with Transfusion-Dependent β-Thalassemia Enrolled in Phase 3 Studies

Janet L. Kwiatkowski, Franco Locatelli, Mark C. Walters, John B. Porter, Suradej Hongeng, Evangelia Yannaki, Andreas E. Kulozik, Martin G. Sauer, Adrian J. Thrasher, Isabelle Thuret, Ashutosh Lal, Ruiting Guo, Richard A. Colvin, Katiana Gruppioni, Alexis A. Thompson

Blood (2021) 138 (Supplement 1): 3085.

DOI: https://doi.org/10.1182/blood-2021-150392

Published: 2021

Journal Articles

Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)

Blood (2021) 138 (Supplement 1): 561.

DOI: https://doi.org/10.1182/blood-2021-147760

Published: 2021

Journal Articles

Rurioctocog Alfa Pegol Use in Immune Tolerance Induction: Interim Results from an Open-Label Multicenter Clinical Trial in Previously Untreated Patients with Severe Hemophilia a

Robert F. Sidonio, Jr., Alexis A. Thompson, Flora Peyvandi, Canan Albayrak, Seoh Leng Yeoh, Joyce Lam, Caterina Maggiore, Werner Engl, Geoffrey Allen, Srilatha Tangada

Blood (2021) 138 (Supplement 1): 3185.

DOI: https://doi.org/10.1182/blood-2021-146143

Published: 2021

Journal Articles

Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence after Treatment with Betibeglogene Autotemcel Gene Therapy: Results from up to 7 Years of Follow-up

Alexis A. Thompson, Franco Locatelli, Evangelia Yannaki, Mark C. Walters, John B. Porter, Suradej Hongeng, Andreas E. Kulozik, Martin G. Sauer, Adrian J. Thrasher, Isabelle Thuret, Ashutosh Lal, Marina Cavazzana, John E.J. Rasko, Lin Du, Richard A. Colvin, Janet L. Kwiatkowski

Blood (2021) 138 (Supplement 1): 573.

DOI: https://doi.org/10.1182/blood-2021-148177

Published: 2021

Journal Articles

Sustained Improvements in Patient-Reported Quality of Life up to 24 Months Post-Treatment with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Mark C. Walters, John F. Tisdale, Markus Y. Mapara, Lakshmanan Krishnamurti, Janet L. Kwiatkowski, Banu Aygun, Kimberly A. Kasow, Stacey Rifkin-Zenenberg, Jennifer Jaroscak, Diana Garbinsky, Costel Chirila, Meghan E. Gallagher, Xinyan Zhang, Pei-Ran Ho, Alexis A. Thompson, Julie Kanter

Blood (2021) 138 (Supplement 1): 7.

DOI: https://doi.org/10.1182/blood-2021-146905

Published: 2021

Journal Articles

Effects of the COVID-19 Pandemic on Caregivers of Young Children with Sickle Cell Disease Enrolled in the Engage-HU Trial

Blood (2021) 138 (Supplement 1): 1891.

DOI: https://doi.org/10.1182/blood-2021-152722

Published: 2021

Journal Articles

Cedar Trial in Progress: A First in Human, Phase 1/2 Study of the Correction of a Single Nucleotide Mutation in Autologous HSCs (GPH101) to Convert HbS to HbA for Treating Severe SCD

Julie Kanter, John F. DiPersio, Patrick Leavey, David C. Shyr, Alexis A Thompson, Matthew H. Porteus, Allison Intondi, Premanjali Lahiri, Daniel P. Dever, Alexandria Petrusich, Joshua Lehrer-Graiwer

Blood (2021) 138 (Supplement 1): 1864.

DOI: https://doi.org/10.1182/blood-2021-152892

Published: 2021

Journal Articles

Metformin for Treatment of Cytopenias in Children and Young Adults with Fanconi Anemia

Blood (2021) 138 (Supplement 1): 1102.

DOI: https://doi.org/10.1182/blood-2021-153598

Published: 2021

Journal Articles

Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers

Mark C. Walters, MD, David H.K. Chui, MD FRCPC, John J Farrell, PhD, Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, John B. Porter, MA, MD, FRCP, FRCPath, Martin G. Sauer, MD, Isabelle Thuret, MD, Suradej Hongeng, MD, Andreas E. Kulozik, MD PhD, Adrian J. Thrasher, MBBS, PhD FMedSci, Evangelia Yannaki, MD, Julia Yang, Dustin Whitney, PhD, Alexandria Petrusich, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD

Blood (2020) 136 (Supplement 1): 1–3.

DOI: https://doi.org/10.1182/blood-2020-137642

Published: 2020

Journal Articles

Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia

Alexis A. Thompson, MD, Janet L. Kwiatkowski, MD MSCE, John B. Porter, MA, MD, FRCP, FRCPath, Suradej Hongeng, MD, Evangelia Yannaki, MD, Andreas E. Kulozik, MD PhD, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Ashutosh Lal, MD, Ruiting Guo, MS, Weijian Liu, PhD, Richard A. Colvin, MD PhD, Mark C. Walters, MD, Franco Locatelli, MD PhD

Blood (2020) 136 (Supplement 1): 52–54.

DOI: https://doi.org/10.1182/blood-2020-135857

Published: 2020

Journal Articles

Health-Related Quality of Life Outcomes for Patients with Transfusion-Dependent Beta-Thalassemia Treated with Luspatercept in the Believe Trial

Maria Domenica Cappellini, Ali T. Taher, Antonio Piga, MD, Farrukh Shah, Ersi Voskaridou, Vip Viprakasit, John B. Porter, MA, MD, FRCP, FRCPath, Olivier Hermine, Ellis J. Neufeld, Alexis A. Thompson, MD, Derek Tang, Peiwen Yu, Shien Guo, Jeevan K. Shetty, Dimana Miteva, Tatiana Zinger, Jay T. Backstrom, Esther Natalie Oliva, MD

Blood (2020) 136 (Supplement 1): 8–9.

DOI: https://doi.org/10.1182/blood-2020-136312

Published: 2020

Journal Articles

Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Julie Kanter, MD, John F. Tisdale, MD, Markus Y Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Ren Chen, Meghan E. Gallagher, MSc, Yuchen Ding, PhD, Sunita Goyal, MD, Clark Paramore, MPH, Alexis A. Thompson, MD, Mark C. Walters, MD

Blood (2020) 136 (Supplement 1): 10.

DOI: https://doi.org/10.1182/blood-2020-136193

Published: 2020

Journal Articles

Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Alexis A. Thompson, MD, Mark C. Walters, MD, Markus Y Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Banu Aygun, MD, Kimberly A. Kasow, DO, Stacey Rifkin-Zenenberg, DO, Manfred Schmidt, PhD, Jay DelCarpini, Francis J. Pierciey, Jr., Alexandra L. Miller, Meghan E. Gallagher, MSc, Ren Chen, Sunita Goyal, MD, Julie Kanter, MD, John F. Tisdale, MD

Blood (2020) 136 (Supplement 1): 16–17.

DOI: https://doi.org/10.1182/blood-2020-134940

Published: 2020

Journal Articles

Addressing Recruitment Challenges in the Engage-HU Trial in Young Children with Sickle Cell Disease

Blood (2020) 136 (Supplement 1): 26–27.

DOI: https://doi.org/10.1182/blood-2020-141471

Published: 2020

NARROW

Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials

Return Visit Rates after an Emergency Department Treat-and-Release Visit for Children with Sickle Cell Pain Episodes

Lovo-cel (bb1111) Gene Therapy for Sickle Cell Disease: Updated Clinical Results and Investigations into Two Cases of Anemia from Group C of the Phase 1/2 HGB-206 Study

Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia

Optimizing Shared Decision Making about Hydroxyurea in Young Children with Sickle Cell Anemia

Relationship of Hydroxyurea Adherence to Depression and Fatigue Among Children and Adolescents with Sickle Cell Disease: A Longitudinal Cohort Study

Improvement in Health-Related Quality of Life Following Treatment with Betibeglogene Autotemcel in Patients with Transfusion-Dependent β-Thalassemia Enrolled in Phase 3 Studies

Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)

Rurioctocog Alfa Pegol Use in Immune Tolerance Induction: Interim Results from an Open-Label Multicenter Clinical Trial in Previously Untreated Patients with Severe Hemophilia a

Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence after Treatment with Betibeglogene Autotemcel Gene Therapy: Results from up to 7 Years of Follow-up

Sustained Improvements in Patient-Reported Quality of Life up to 24 Months Post-Treatment with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Effects of the COVID-19 Pandemic on Caregivers of Young Children with Sickle Cell Disease Enrolled in the Engage-HU Trial

Cedar Trial in Progress: A First in Human, Phase 1/2 Study of the Correction of a Single Nucleotide Mutation in Autologous HSCs (GPH101) to Convert HbS to HbA for Treating Severe SCD

Metformin for Treatment of Cytopenias in Children and Young Adults with Fanconi Anemia

Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers

Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia

Health-Related Quality of Life Outcomes for Patients with Transfusion-Dependent Beta-Thalassemia Treated with Luspatercept in the Believe Trial

Improvements in Health-Related Quality of Life for Patients Treated with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Addressing Recruitment Challenges in the Engage-HU Trial in Young Children with Sickle Cell Disease

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